Abstract

CURRENT AND EMERGING TREATMENT FOR PAROXYSMAL NOCTURNAL HEMOGLOBINURIA, WITH EMPHASIS ON THE COMPLEMENT INHIBITOR IPTACOPAN AND DANICOPAN

A. S. Nikitha* and Bhavana Manjunath Bhat

Abstract

Paroxysmal nocturnal haemoglobinuria (PNH) is a life-threatening and rare haematological disease that is defined by the intravascular haemolysis due to the autoimmune system mediated destruction of the red blood cells causing some of the symptoms of fatigue, impotence and thrombosis. Until recently, treatments for PNH were restricted and the usual management of PNH include using anticomplement component 5 (C5) monoclonal antibodies like eculizumab or ravulizumab with a primary aim of controlling symptoms and averting threat. Though, there exists newer drugs such as iptacopan and danicopan which is a new, oral complement factor B inhibitor and factor D inhibitors respectively, for PNH patients.

Keywords: Iptacopan, Danicopan, Paroxysmal nocturnal haemoglobinuria (PNH).